Gene Therapy Trial for Stargardt Disease Reaches Full Enrollment

This article was originally published on our sister site, Opthalmology Times.

Nanoscope Therapeutics Inc. today announced full enrollment of its Phase 2 clinical trial of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for Stargardt disease. Six-month data from the Phase 2 STARLIGHT trial are expected in H1 2023.

"We are excited by the therapeutic potential of MCO-010, supported by robust data from previous preclinical and clinical studies," Sulagna Bhattacharya, CEO of Nanoscope, said in a statement. "Completing the quick two-month enrollment of this Phase 2 trial, which will evaluate the safety and effects of a single intravitreal injection of MCO-010, brings us another major step forward in developing this novel therapy with broad therapeutic application."

The Phase 2 STARLIGHT open-label trial (NCT05417126) enrolled 6 subjects with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease. In this study, all subjects received the same single intravitreal dose of 1.2E11gc/eye of MCO-010 as used in the Company's Phase 2b retinitis pigmentosa (RP) study.

"Stargardt disease is a blinding condition affecting adults and children alike. It is the most common macular dystrophy and has no established treatment," said Byron Lam, MD, Robert Z. & Nancy J. Greene Chair and a professor of Ophthalmology at the University of Miami and principal investigator in the Phase 2 STARLIGHT trial. "MCO-010 has the potential to improve visual function in RP and Stargardt patients, and we look forward to seeing the data from this study."

Nanoscope's optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells to enable vision in different color environments. The therapy is administered as a single intravitreal injection for in-office delivery without the need for any other devices or interventions. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA and is concurrently being evaluated in Nanoscope's Phase 2b RESTORE trial in patients with RP, with trial results also expected in H1 2023.